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20 years of gene therapy for SCID

Nature Immunology volume 11, pages 457–460 (2010)Cite this article

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Severe combined immunodeficiency conditions are devastating disorders of adaptive immunity. Although these diseases were initially treated by transplantation of allogeneic hematopoietic stem cells, the past 20 years has shown that these conditions are correctable by gene therapy.

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Figure 1: Block in lymphocyte differentiation resulting from a deficiency in γc (SCID-X1) and ADA.
Figure 2: Gene therapy for SCID-X1 and ADA deficiency.

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Acknowledgements

We thank many colleagues who have taken part in this research, notably G. de Saint Basile, J. Di Santo, A. Durandy, C von Kalle and R. Bushman; A. Aiuti, F. Candotti, D.B. Kohn and A. Thrasher for providing unpublished data on their ongoing research; and the many colleagues who have contributed to the field whose important publications could not be cited here because of space constraints.

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Authors and Affiliations

  1. Alain Fischer is with the Institut National de la Santé et de la Recherche Médicale, Paris, France,

    Alain Fischer

  2. Paris-Descartes University, Paris, France

    Alain Fischer, Salima Hacein-Bey-Abina & Marina Cavazzana-Calvo

  3. Unité d'Immunologie et Hématologie Pédiatrique, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants Malades, Paris, France

    Alain Fischer

  4. Salima Hacein-Bey-Abina and Marina Cavazzana-Calvo are with the Institut National de la Santé et de la Recherche Médicale, Paris, France,

    Salima Hacein-Bey-Abina & Marina Cavazzana-Calvo

  5. Department of Biotherapy, Centre d'Investigation Clinique intégré en Biothérapies, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France

    Salima Hacein-Bey-Abina & Marina Cavazzana-Calvo

Authors
  1. Alain Fischer
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Correspondence to Alain Fischer.

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Fischer, A., Hacein-Bey-Abina, S. & Cavazzana-Calvo, M. 20 years of gene therapy for SCID. Nat Immunol 11, 457–460 (2010). https://doi.org/10.1038/ni0610-457

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